| Term | Definition |
|---|
| Gene Therapy | Intracellular delivery of genetic material to generate a therapeutic effect by correcting an existing abnormality or providing cells with a new function |
| What is the application for gene therapy? | Inherited genetic disorders and acquired diseases |
| Human Gene Therapy Trials mostly study what disease? | Cancer |
| Jesse Gelsinger, first person to die in a clinical trial for gene therapy in 1999, suffered from what? | Ornithine transcarbamylase deficiency |
| What are the two types of Gene Therapy Cells? | Somatic and Germline |
| Which Gene Therapy Cell type is used for stem cell, fetal, and xenotransplantation gene therapy? | Somatic |
| Which Gene Therapy Cell type is used for cloning? | Germline |
| Developmental biologist James Thompson is known for what? | He derived the first embryonic stem cell lines from frozen embryos |
| Dolly the sheep was clones by what two types of cells implanted in a surrogate mother? | Egg and Somatic cell |
| True or False: Stem cells can create any type of cell, tissue, or organ? | True |
| In ethics for animal cloning, what are the pros? | Better research capabilities, producing biological proteins, better livestock, and regenerating organs |
| In ethics for animal cloning, what are the cons? | Decline in genetic diversity, immature techniques, and destroying life |
| What types of cells are used in plant cloning? | Totipotent Cells |
| Which type of Gene Therapy cells can be passed on to future progeny? | Germline |
| How Gene Therapy Works: ___________ a mutated gene | Correct |
| How Gene Therapy Works: ____________ a defective or missing gene | Replace |
| How Gene Therapy Works: ____________ the functions of the genes present | Augment |
| How Gene Therapy Works: ___________ a specified sensitivity to a normally inert prodrug | Instill |
| How Gene Therapy Works: ___________ the target gene expression to restore cell or tissue functions | Block |
| How Gene Therapy Works: ___________ with the life cycle of infectious diseases | Interfere |
| What is the " therapeutic gene based" gene therapy approach? | administration of a gene delivery system (normally by injection) |
| What is the "Cell based" gene therapy approach? | transplanted with stem cells or genetically engineered cells |
| What is the "Tissue/organ based" gene therapy approach? | transplanted with genetic differentiated or engineered tissue or organs |
| What is the pH of a lysosome? | 4 to 5 |
| What is the pore size of the nucleus? | 2.5 micrometer |
| What is "in vivo" gene delivery? | Injection for systemic delivery |
| What is "ex vivo" gene delivery? | Take genes out of body, manipulate, then re-implanted into body |
| What is "In situ" gene delivery? | Local delivery injected directly into desired tissue |
| Transcription take place in the? | nucleus |
| Transcription transcribes ____ into ____ | DNA, mRNA |
| Translation in the cytosol translates ______ into _______ to make ______ | mRNA, tRNA, protein |
| What is a gene carrier? | A vehicle that safely delivers nucleic acids to target cells |
| Virus vector production involves taking out the _______ DNA and replaced with the __________ gene | Viral, Therapeutic |
| What are the Extracellular barriers to gene delivery? | Opsonin, phagocytes, enzymes and serum proteins |
| What are the Intracellular barriers to gene delivery? | Cell membrane, endosomal membrane, cytoplasmic transport, and the nuclear membrane |
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